Rare Disease

Five things for pharma marketers to know: Friday, May 1

Five things for pharma marketers to know: Friday, May 1

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Nexium tops the list of Medicare's spending on brand drugs; Gilead Sciences doubles its first-quarter profit; Pfizer reportedly wants to buy a rare-disease drug company

Rituxan may have rare-disease use

The cancer and immunology drug may help patients with a condition that affects two people in every one million.

Horizon makes $1.1-billion rare-disease push

Horizon makes $1.1-billion rare-disease push

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Horizon Pharma's purchase of Hyperion Therapeutics will add two rare-disease drugs to its portfolio.

Shire and Cincinnati Children's Hospital team up in rare diseases

Shire and Cincinnati Children's Hospital team up in rare diseases

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The duo hope to begin testing a new treatment within three years.

Everyday Health buys rare-disease agency Cambridge BioMarketing

Everyday Health buys rare-disease agency Cambridge BioMarketing

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The $38-million deal expands the reach of rare-disease campaigns.

BI rare-disease awareness push slated for Saturday

Industry rare disease awareness efforts included a pipeline overview at Pfizer as well as BI's efforts to draw attention to IPF.

NPS drug approval bolsters Shire acquisition decision

NPS drug approval bolsters Shire acquisition decision

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The FDA approved NPS Pharmaceuticals's rare-disease drug Friday. The company, which Shire shelled out $5.2 billion to buy, expects it to hit the market during the second-quarter.

Mallinckrodt plans expansion of commercial staff

The specialty biopharma firm says it's hiring new commercial managers after a buying spree.

Five things for pharma marketers to know: Monday, November 24

Five things for pharma marketers to know: Monday, November 24

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BioMarin to acquire Dutch biotech Prosensa; Woodcock says significant efficacy key to Breakthrough Therapy designation; Stryker considers $16 billion bid for UK devicemaker Smith and Nephew.

Rethinking Healthcare Communications Through the Prism of Rare Diseases

Rethinking Healthcare Communications Through the Prism of Rare Diseases

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Success today is predicated on recognizing that every patient is different, every disease is different, every drug, every breakthrough and every cure is different.

Sanofi prices Cerdelga

Sanofi prices Cerdelga

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The $310,000 price for the Gaucher disease medication is typical of the rare-disease category.

FDA wants Alexion to clean up

FDA inspectors have cited the manufacturer of the rare-disease drug Soliris (eculizumab) for poor manufacturing practices.

Sanofi expands Gaucher disease portfolio

Sanofi expands Gaucher disease portfolio

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The oral medication Cerdelga joins Cerezyme in its Gaucher disease arsenal. Sanofi expects the drug will be priced "on par" with Cerezyme, which goes for around $300,000 a year in the US.

Amicus seeks to upset Fabry market

Amicus seeks to upset Fabry market

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Phase-III tests indicate patients may be able to switch from injectable enzyme-replacement therapies, like Fabrazyme, to the firm's oral drug.

Shire expands rare disease reach

A $225-million deal with US biotech ArmaGen gives Shire worldwide commercialization rights to an experimental enzyme replacement therapy.

Advocacy group alleges Sanofi-Shire conspiracy

An advocacy group alleges that Shire's decision to ignore the US Fabry disease market was the result of a backroom deal with Sanofi.

Aegerion shows rare disease not slam dunk

Aegerion shows rare disease not slam dunk

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Analyst reaction to this week's quarterly news reflected a need to woo investors, adding to some negative carryover from the previous year.

Sarepta plans for 2015 drug review

Sarepta plans for 2015 drug review

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The company said it plans to file its rare-disease drug with the FDA later this year, for a 2015 review.

Amgen's PSCK9 shows rare disease potential

Amgen's PSCK9 shows rare disease potential

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The Thousand Oaks, Calif. company says its cholesterol-lowering antibody hit its clinical trial endpoint among rare disease patients.

NICE wants more Soliris information

The UK watchdog wants to know what's behind the rare-disease drug's high price.

Sanofi seeks to revive Kynamro launch

Sanofi seeks to revive Kynamro launch

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Kynamro's slow start, coupled with competition, has prompted Sanofi to add sales reps.

Rare diseases may be less so

Regulatory Focus discusses how the expanding US population may make it harder for a condition to qualify as rare.

Genzyme rare-disease drug gets priority label

The FDA granted priority review for the experimental, all-oral Gaucher's disease treatment.

FDA pushes review date for Biogen blood drug

The agency extended the review time for Biogen's Alprolix by three months.

FDA panel backs BioMarin rare-disease drug

FDA panel backs BioMarin rare-disease drug

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The patient population is small, at around 3,000, but the expected price tag is $330,000 a year.

IMS predicts $1T global drug market by 2017

IMS predicts $1T global drug market by 2017

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The catch is the difference between sales and revenues. Generics will continue to be a force, and small-audience drugs will become increasingly important.

Alexion recalls some Soliris batches

The US-only recall amounts to less than 1% of the company's inventory.

FDA delays Sarepta MD drug

The regulator says the drugmaker needs to do more testing.

Shire, Salix buy way to expansion

The companies are plunking down substantial sums to increase their reach.

Lilly partners with NIH for rare-disease research

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Eli Lilly is the first Big Pharma to team up with the agency in its rare-disease pre-clinical development program.

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